Mark Pruzanski, MD – CEO & Founder
Roberto Pellicciari, Ph.D. – Director of Medicinal Chemistry & Scientific Founder
Stefano Fiorucci, MD – Director of NR Biology & Scientific Founder
Steve Kliewer, Ph.D. – Director of NR Biology & Scientific Founder

Background

Intercept Pharmaceuticals, Inc. is a New York City-based specialty pharmaceutical company focused on developing small molecule drugs for the treatment of chronic liver and metabolic disorders. Intercept’s lead compound INT 747, a small molecule nuclear receptor, was discovered in 2001 through collaboration between GlaxoSmithKline and University of Perugia scientists. Originally the compound was pushed aside by Big Pharma as it was viewed too small an opportunity to pursue. In 2002 Chief Executive Officer Mark Pruzanski, MD, a native New York physician, stepped in and Intercept was formed.

Since its inception, Intercept has functioned as a truly virtual organization with 15 professionals formally associated with the company conducting activities in the US and Europe. The company has focused efforts on indications that have no current treatment such as alcoholic liver disease, hepatitis C, and NASH (which is related to obesity). Pruzanski believes the tens-of-millions of patients with no alternatives, except organ transplant, will help turn INT 747 into a blockbuster.

The company is currently involved in numerous collaborations including work with Scott Friedman, MD, a well-known liver disease researcher at New York’s Mt. Sinai, and a recently inked collaboration with researchers at the University at Colorado investigating the action of FXR on kidney dysfunction.

To date, Intercept has been successful in securing approximately $4.5 million to support activities within the company. Some well-known financial groups have contributed to Intercept’s pockets including Joseph Stevens & Company, Rodman & Renshaw and Brookstone Biotech Ventures. The company has managed the funds wisely and is on target regarding their milestones; they’re currently putting together the first IND for INT 747.

The Technology

Intercept’s INT-747 (formerly 6ECDCA) is a potent and selective semisynthetic FXR (farnesoid X receptor) agonist offering a novel approach to the treatment of fibrotic and cholestatic chronic liver diseases, as well as other GI and renal diseases. In gold standard rodent models, human hepatocyte stellate cell lines, activation of FXR has been shown to both dramatically inhibit the synthesis of fibrogenic collagen and enhance fibrolytic resolution of cirrhosis. INT-747 is an analog of CDCA, a natural bile acid that has long been used as a human therapeutic and more recently was identified as the primary physiologic FXR ligand. Intercept’s patent pending lead compound is a >100 fold more potent FXR agonist than CDCA.

Looking Ahead

According to Pruzanski, there exists definite plans to build out the infrastructure of Intercept but he does believe that the virtual model that has been utilized will continue to work for them. His intention is to keep the company’s headquarters in New York City in addition to the current research facilities in Italy.

With respect to financing, Intercept is currently targeting a $10-15 million Series A round. Pruzanski believes that this round of funding will allow Intercept to take INT 747 through a phase II proof-of-concept as well as an IND for a second indication by the end of 2007. Additionally, he stated that the company will be on the lookout for appropriate in-licensing opportunities in parallel to what is being created internally.

Contact information: Mark Pruzanski, MD CEO, Intercept Pharmaceuticals T 917 744 4043 E mark@interceptpharma.com.